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Reshaping Clinicians' Perceptions of Disease Burden in Becker Muscular Dystrophy: The Impacts of Incorporating the Patient Perspectives in CME

Author:Drexel, Carole   Wilson, Eve   Veerapandiyan, Aravindhan   Bello, Luca   Kowalski, Katie   

Session Name:P2: Education, Research, and Methodology: Neurohumanities and Education  

Topic:Education, Research, and Methodology  

Program Number:P2.005  

Author Institution:PlatformQ LLC, Needham, MA  PlatformQ LLC, Bowie, MD  Arkansas Childrens Hospital/UAMS, Little Rock, AR  University of Padova, Department of Neuroscience NPSRR, Padova, Italy  National Organization for Rare Disorders, Washington, DC  

RGX-202, An Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Phase I/II Clinical Data

Author:Veerapandiyan, Aravindhan   Harper, Amy   Iannaccone, Susan   Kuntz, Nancy   Tesi Rocha, Ana Carolina   Boulos, Nidal   Wilson, Catherine   Tsao, Elisa   Gilmor, Michelle   Curtiss, Stacey   Patel, Hiren   Fiscella, Michele   Danos, Olivier   Pakola, Stephen   Dastgir, Janannaz   

Session Name:P8: Child Neurology and Developmental Neurology: Neuromuscular Disorders 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:P8.005  

Author Institution:Arkansas Childrens Hospital/UAMS, Little Rock, AR  Children’s Hospital of Richmond at Virginia Commonwealth University, Richmond, VA  Department of Pediatrics, Dallas, TX  Ann & Robert H Lurie Children'S Hospital of Chicago, Chicago, IL  Stanford University, PALO ALTO, CA  REGENXBIO, Rockville, MD  Regenxbio, Durham, NC  Regenxbio Inc, Rockville, MD  Regenxbio, Irvington, NY